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Table 2 PROM scores: summary of Parts 1, 2, and 3

From: Development and evaluation of a patient-reported outcome measure specific for Gaucher disease with or without neurological symptoms in Japan

Part

Pre-test

Main analysis

GD1

(N = 3)

GD2

(N = 6)

GD3

(N = 7)

Overall

(N = 16)

GD1

(N = 9)

GD2

(N = 13)

GD3

(N = 11)

Overall

(N = 33)

Part 1

 Mean (SD)

NA

27.5 (10.6)

38.8 (27.2)

35.0 (22.4)

42.1 (31.5)

13.8 (12.4)

65.0 (21.3)

45.0 (30.3)

 Median

(min, max)

NA

27.5

(20.0, 35.0)

30.0

(17.5, 77.5)

28.8

(17.5, 77.5)

37.5

(10.0, 97.5)

13.8

(5.00, 22.5)

65.0

(40.0, 90.0)

42.5

(5.00, 97.5)

 Not collected, n (%)

3 (100)

4 (66.7)

3 (42.9)

10 (62.5)

3 (33.3)

11 (84.6)

7 (63.6)

21 (63.6)

Part 2

 Mean (SD)

17.0 (8.19)

45.8 (24.1)

16.2 (15.7)

25.5 (21.4)

17.0 (8.47)

38.7 (21.5)

26.5 (20.6)

29.1 (20.3)

 Median

(min, max)

15.0

(10.0, 26.0)

51.0

(12.0, 69.0)

8.00

(5.00, 45.0)

15.0

(5.00, 69.0)

17.0

(7.00, 28.0)

48.0

(7.00, 76.0)

28.0

(0, 67.0)

27.0

(0, 76.0)

 Not collected, n (%)

0 (0)

2 (33.3)

1 (14.3)

3 (18.8)

1 (11.1)

0 (0)

0 (0)

1 (3.0)

Part 3

 Mean (SD)

NA

79.0 (41.4)

25.7 (12.7)

52.3 (40.0)

24.2 (16.5)

79.7 (42.5)

52.1 (30.2)

57.9 (39.6)

 Median

(min, max)

NA

95.0

(32.0, 110)

33.0

(11.0, 33.0)

33.0

(11.0, 110)

21.0

(5.00, 52.0)

94.5

(0, 126)

51.5

(13.0, 109)

52.5

(0, 126)

 Not collected, n (%)

3 (100)

3 (50.0)

4 (57.1)

10 (62.5)

3 (33.3)

1 (7.7)

1 (9.1)

5 (15.2)

  1. GD1/2/3: type 1/2/3 Gaucher disease; max: maximum; min: minimum; NA: not applicable; PROM: patient-reported outcome measure; SD: standard deviation